A first-of-its-kind stem cell-based retinal implant for patients with advanced dry age-related macular degeneration was well-tolerated and showed promising early efficacy in a phase 1/2a study.

Researchers evaluated the use of human embryonic stem cells implanted in patients with advanced dry AMD. The study evaluated the primary safety of the treatment and noted any restoration of vision or prevention of vision loss in a patient cohort, lead author Amir H. Kashani, MD, PhD, assistant professor of clinical ophthalmology at the Keck School of Medicine of USC, told Healio.com/OSN.

“The idea of the study is we can use human embryonic stem cells that are differentiated into mature adult RPE (retinal pigment epithelium) cells and to replace the area of RPE loss in geographic atrophy. By doing that, we can presumably restore some vision or prevent further vision loss. The two unique things of the study are that we are replacing the RPE cells as a single sheet and in the area of actual RPE loss,” he said.

A stem cell-based retinal implant for patients with advanced dry age-related macular degeneration was well tolerated in a phase 1/2a study.

Source: Adobe images

One-year study

The patients were followed for up to 1 year in this initial study, Kashani said.

No serious adverse events related to the implant or the same-day surgery were reported. The treatment was well-tolerated by patients in the cohort, and the implant integrated with the patients’ retinal tissue, he said.

Of the first five subjects in the cohort, one experienced a 17-letter improvement in visual acuity at day 60 after the implant. The improvement was maintained through day 120, according to the study data.

Two patients experienced improvements in visual function and could visually fixate on a specific location, indicating improved function of the retina, Kashani said.

Encouraging results

The results of the study were encouraging, considering the enrolled patients had chronic disease. This was primarily a safety study, so the researchers were not necessarily expecting big improvements in visual function, Kashani said.

“We’re focusing on finishing this trial and doing a comprehensive assessment of the preliminary efficacy and safety. The next immediate goal is that phase 2 study, which will most likely be a longitudinal study comparing implantation subjects with a standard of care, which is no treatment at this point in other patients, to see if it truly does prevent vision loss or restore vision in the patients. That will be very important for us to be able to assess when it works, how it works and even why it works,” he said.

The study was funded in part by the California Institute for Regenerative Medicine. – by Robert Linnehan

Disclosure: Kashani reports he receives speaking fees, grants and honoraria from Carl Zeiss Meditec and is on an advisory board for Alimera Sciences.

A first-of-its-kind stem cell-based retinal implant for patients with advanced dry age-related macular degeneration was well-tolerated and showed promising early efficacy in a phase 1/2a study.

Researchers evaluated the use of human embryonic stem cells implanted in patients with advanced dry AMD. The study evaluated the primary safety of the treatment and noted any restoration of vision or prevention of vision loss in a patient cohort, lead author Amir H. Kashani, MD, PhD, assistant professor of clinical ophthalmology at the Keck School of Medicine of USC, told Healio.com/OSN.

“The idea of the study is we can use human embryonic stem cells that are differentiated into mature adult RPE (retinal pigment epithelium) cells and to replace the area of RPE loss in geographic atrophy. By doing that, we can presumably restore some vision or prevent further vision loss. The two unique things of the study are that we are replacing the RPE cells as a single sheet and in the area of actual RPE loss,” he said.

A stem cell-based retinal implant for patients with advanced dry age-related macular degeneration was well tolerated in a phase 1/2a study.

Source: Adobe images

One-year study

The patients were followed for up to 1 year in this initial study, Kashani said.

No serious adverse events related to the implant or the same-day surgery were reported. The treatment was well-tolerated by patients in the cohort, and the implant integrated with the patients’ retinal tissue, he said.

Of the first five subjects in the cohort, one experienced a 17-letter improvement in visual acuity at day 60 after the implant. The improvement was maintained through day 120, according to the study data.

Two patients experienced improvements in visual function and could visually fixate on a specific location, indicating improved function of the retina, Kashani said.

Encouraging results

The results of the study were encouraging, considering the enrolled patients had chronic disease. This was primarily a safety study, so the researchers were not necessarily expecting big improvements in visual function, Kashani said.

“We’re focusing on finishing this trial and doing a comprehensive assessment of the preliminary efficacy and safety. The next immediate goal is that phase 2 study, which will most likely be a longitudinal study comparing implantation subjects with a standard of care, which is no treatment at this point in other patients, to see if it truly does prevent vision loss or restore vision in the patients. That will be very important for us to be able to assess when it works, how it works and even why it works,” he said.

The study was funded in part by the California Institute for Regenerative Medicine. – by Robert Linnehan